Clinical trials aim to evaluate the effectiveness and safety for patients of a medical intervention, based on comparable results. To determine and compare clinical safety and efficacy, clinical trial results must reflect relevant ‘outcome measures’. These outcomes should reflect what must be measured and reported in clinical trials in specific disease areas. Regulatory agencies, standards organisations and international societies have issued a number of guidance documents on outcomes, but many trials, on rare diseases in particular, still do not include standardised outcomes in clinical data. Overall, insufficient attention to the selection of clinical trial outcomes has led, too frequently, to a waste of generated data, research efforts, and inefficiencies in drug development and in regulatory review processes. This area has been identified by IRDiRC’s Scientific Committees as an area of focus; consequently a Task Force with several nominated experts in the field has been set up.
This Task Force organizes a discussion on Patient-Centred Outcome Measures to identify what should and can be done to support the development of patient-relevant outcome measures for rare diseases, in order to improve the feasibility and quality of forthcoming trials and to provide data of relevance to the patient community and other decision makers. IRDiRC’s Task Force and the Scientific Secretariat have made a background document available that forms the starting point for debate, which is currently open for comments and suggestion for the community at large.
More information about this Task Force can be found here.